Researchers develop new gene repair technique

The team said that the novel technique is much simpler than previous methods and establishes the groundwork for major advances in regenerative medicine, drug screening and biomedical research.

The new technique targets and repairs defective genes using human pluripotent stem cells and DNA-cutting protein from meningitis bacteria.

Singapore: Researchers from the Morgridge Institute for Research and Northwestern University have created an efficient way to target and repair defective genes, using human pluripotent stem cells and DNA-cutting protein from meningitis bacteria.

In a report published in Proceedings of the National Academy of Sciences, the team said that the novel technique is much simpler than previous methods and establishes the groundwork for major advances in regenerative medicine, drug screening and biomedical research.

Zhonggang Hou of the Morgridge Institute's regenerative biology team and Yan Zhang of Northwestern University served as first authors on the study; James Thomson, director of regenerative biology at the Morgridge Institute, and Erik Sontheimer, professor of molecular biosciences at Northwestern University, served as principal investigators.

"With this system, there is the potential to repair any genetic defect, including those responsible for some forms of breast cancer, Parkinson's and other diseases," Hou said. "The fact that it can be applied to human pluripotent stem cells opens the door for meaningful therapeutic applications," he added.

Zhang said the Northwestern University team focused on Neisseria meningitidis bacteria because it is a good source of the Cas9 protein needed for precisely cleaving damaged sections of DNA.